CAR-T therapies, a niche opportunity to fight cancer

The treatment of certain types of cancer has been revolutionised by CAR-T therapies (Chimeric Antigen Receptor T cell). This is an innovative approach to gene therapy, as it involves genetically modifying the patient’s immune system cells to make them more effective in the fight against cancer.

This therapy aims to make the human body’s T-cells fight cancer by altering them in the laboratory so that they can act as agents that seek out and destroy cancer cells.  In this way, they can treat certain types of leukaemias and lymphomas in particular.

These therapies can reverse the situation in at least 30% of patients in whom a blood tumour would have wiped out any chance of life. CAR-T cells are the last, and revolutionary, missing piece in the cancer-fighting arsenal. CAR-T cell therapy is revolutionising the prognosis and life expectancy of patients with some types of cancer, and its journey has only just begun.

However, this new generation of personalised immunotherapies also poses new healthcare, regulatory, and financial challenges, which it is desirable to anticipate to optimise their approach within the healthcare system. Advanced therapies are a 21st-century innovation that must be implemented in a healthcare system with 20th-century organisational, human resources, accounting, and management structures.

For this reason, implementing an innovation of the scale of CAR-T is a considerable challenge for the National Health System, for its professionals, and also for patients and their families, and the implementation of CAR-T therapies has opened the way for new models of both research and funding at European and international level.

How does CAR-T therapy work?

T-cell collection: T-cells are removed from the patient through a process called leukopheresis. These T cells are a type of white blood cell that plays a crucial role in the body’s immune response.

Genetic modification: In the laboratory, harvested T cells are genetically modified to express a chimeric antigen receptor (CAR). This receptor is specifically designed to recognise and bind to antigens present on cancer cells.

Cell expansion: once modified, T cells are cultured in large numbers in the laboratory to generate a sufficient population of CAR-T cells.

Patient preparation: while the CAR-T cells are being produced, the patient undergoes conditioning therapy that may include low-intensity chemotherapy to eliminate some of the existing immune systems and allow the CAR-T cells better access to the tumour.

CAR-T cell infusion: Once CAR-T cells have been cultured in sufficient quantity, they are administered to the patient through an intravenous infusion. These modified cells multiply in the patient’s body and, by recognising the cancer antigens, attack and destroy the malignant cells.

Response and follow-up: after infusion of CAR-T cells, the patient is closely monitored to assess response to treatment, as well as to monitor and manage any side effects that may arise. CAR-T therapies are showing good results in the treatment of certain refractory or recurrent cancers. However, they are experimental therapies that face new challenges, and in some individual patients may have rare negative side effects, such as treatment-associated encephalopathy or cytokine release syndrome.

A niche of opportunities

The Food and Drug Administration (FDA) currently has five CAR-T therapies approved on the market, and there are about a thousand CAR-T clinical trials worldwide. The market for these therapies is dominated by three major companies: US-based Gilead Sciences and Bristol Myers Squibb and Swiss-based Novartis International.

As these therapies have made significant progress in the fight against cancer, research is underway to improve their efficacy and safety and also to extend their application to other types of cancer such as lung, breast and prostate.

Another new niche opportunity lies in exploring the combination of these therapies with other approaches such as gene therapy, immunotherapy or targeted therapy, in order to improve treatment effectiveness and overcome treatment resistance.

The European Cancer Plan published in 2021, in addition to launching 10 flagship initiatives and 32 actions in various policies, proposes funding of more than 4 billion euros between 2021 and 2027 to fight cancer. Horizon Europe will invest more than €2 billion, not only to support basic research to develop new CAR-T targets in Pillar 1 (ERC; MSCA) but also the development of innovative technologies for advanced and sustainable manufacturing of these therapies through the EIC programme in Pillar 3, as well as the first phases of clinical validation of these therapies in citizens through the Pillar 2, Cluster Health and the Mission Cancer programme.

In addition, the EU4Health programme aims to invest more than €1.25 billion in actions to enable health systems to cope with the implementation of these innovative therapies.